To Drive Value in Hematology Research, We Must Prioritize the Patient Experience

Hematology research has accelerated rapidly, making tremendous progress in the first half of this decade. Since 2020, the FDA has approved more than 40 new therapies for blood cancers and rare blood diseases, and over 35 additional new indications for products already on the market.¹ Standards of care continue to improve for certain disease states, including hemophilia and multiple myeloma. For others, such as patients with immune thrombocytopenia (ITP), the treatment paradigm is expanding, offering patients different options for the first time in years.

As with any medical condition that is chronic or requires lifelong care, living with a blood disease or cancer can sometimes come with unavoidable burden: lengthy treatment administration times, adverse effects, frequent dosing, challenges accessing care, or negative long-term health outcomes. For some conditions, there is the potential of an ongoing cycle of relapse and remission.

This doesn’t by any means negate the value of the advancements made to date, but it does reinforce that progress cannot be measured simply by the number of new treatment options available to patients. Clinical research and innovation must also aim to demonstrate the impact of new treatments on the multidimensional needs and experiences of people living with these hematologic conditions. This patient-centric approach is the foundation for our own clinical programs at Sanofi.

Hematology research must consider the entire patient experience, not only clinical outcomes.

For many years, advancements in hematology research have been centered on clinical end points, whether it’s improving bleed protection in hemophilia, addressing disease progression in multiple myeloma, or restoring platelet counts in ITP. Today, we have opportunities to look more deeply at how these advancements impact other aspects of a patient’s disease journey.

We’ve made a point to include health-related quality-of-life (HRQOL) analyses as part of our key hematology studies so that physicians can partner with patients to make more holistic decisions on a given treatment approach based on their individual needs or lifestyle goals. Exploring other QOL end points in depth can offer further insight into how treatments could potentially improve other disease-related complications. We understand that addressing these challenges for patients can also have a broader impact on their communities of caregivers, families, and friends.

We also know that the way medicines are administered can make a meaningful difference throughout the treatment experience. This compels us to study ways to reduce treatment frequency to better fit with a person’s lifestyle needs or simplify treatment administration methods to lift the burden on patients and the providers who care for them. By conducting thoughtfully designed trials, we can generate clear evidence to show how certain approaches can improve patient satisfaction and create impact beyond what’s traditionally achieved in the clinic.

Optimizing insights from other scientific pursuits can offer new avenues of addressing unmet patient needs.

Our approach to hematology research is heavily influenced by our expertise in immunoscience, which looks at the role of the immune system across diseases and how we can optimize these insights to help us treat hematologic conditions. This focus has enabled accelerated learnings in several blood cancers and immune-mediated blood diseases.

Immune pathways previously studied in oncology help inform treatment advancements for immune-mediated blood diseases. Today, these learnings are steering research in other immune-mediated diseases. Following the science could one day yield improvements, not just in disease outcomes, but also in the overall burden on patients.

To truly move the needle on patient experience, real-world evidence is a must.

While the controlled nature of clinical studies is essential for demonstrating safety and efficacy, there are limitations when it comes to more qualitative outcomes or circumstances, particularly in hematology. Real-world evidence can help fill these gaps in a way that reflects the broader patient population and substantiates findings from the clinic.

In hemophilia, there has been an influx of treatment options and modalities.² In this specific scenario, real-world evidence can help inform shared decision-making between patient and physician to identify the right treatment fit based on the individual’s specific lifestyle goals. It also provides a more robust picture of how medicine may affect patients’ quality of life and functional health.

From an industry perspective, real-world data in hematology research can also help optimize clinical trial designs, ensuring our efforts are truly geared toward addressing gaps in care.

If the past 5 years are any indication, the next 5 could bring significant scientific breakthroughs for patients across the broader hematology space. By putting patients at the center of research and innovation, we can drive a holistic paradigm shift in the lived experience of people with rare blood diseases and blood cancers.

Author Information

Tom Snow, MBA, is general manager, US Oncology, and Jeff Schaffnit, MBA, is general manager, US Rare Blood Disorders, both at Sanofi.

References

1. Oncology (cancer)/hematologic malignancies approval notifications. FDA. Updated March 25, 2026. Accessed February 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications.
2. Windyga J. Advances in hemophilia treatment. Acta Haemato Pol. 2025;56(supp S):53-60. doi:10.5603/ahp.10851